Alzheimer’s Program

Alzheimer’s disease (AD) is a brain disease that slowly destroys memory and thinking, and eventually, the ability to carry out very simple tasks. At the current time, there is no cure. AD is the leading cause of dementia (loss of thinking, remembering, and reasoning) and currently affects 12 million people worldwide; 5.4 million of these are in America alone, a figure that is predicted to triple by 2050. Dementia ranges in severity from the mild stage, when it is just beginning to affect a person’s functioning, to the severe stage, when the person must depend completely on others for basic care causing a tremendous burden on the patient and caretakers. Alzheimer’s disease is named after Dr. Alois Alzheimer, who in 1906 noticed changes in the brain tissue of a patient who died of a unique mental illness. Upon examination of the patient’s brain, he found many abnormal clumps (known now as amyloid plaques) and tangled bundles of fibers (known now as neurofibrillary tangles) in a region of the brain called the forebrain. Plaques and tangles in the brain are two of the main features of Alzheimer’s disease. These plaques and tangles accumulate in the neurons and over time kill the cells leading to a progressive loss of neurons. As neurons die, their physiological functions slow down, and the symptoms of AD start to manifest. Over the past decade stem cells have become a viable option for a new therapy for AD. Here at the Neural Stem Cell Institute (NSCI) we use our expertise in neural stem cells to discover novel treatments and therapies for curing and treating patients with AD.

Here are some approaches we take.

1. Cells for Replacement Therapy: Recently it has been shown that skin cells can be turned into a pluripotent stem cell (PSC) through a process called reprogramming. PSCs are unique in that they are capable of turning into any cell type of the human body, including the neurons of the brain. Currently, scientists at NSCI can create these human PSC lines, and then turn them into the forebrains that die in AD. These healthy neurons can be used as a source of cells to implant into an AD patient in hopes of replacing the sick and dying cells.

2. Cells for Drug Discovery: Using the same cell reprogramming technology, scientists at NSCI can derive human PSCs from healthy and AD patients. The healthy PSCs can be manipulated into the neurons that die in AD patients, to get a better understanding of the disease pathway. Having both healthy and diseased neurons from AD patients provides scientist at NSCI a “disease in a dish” model of AD, which can be used to discover novel drugs that prevent the sick neurons from dying.

3. Identifying the Cause of Alzheimer’s Disease: Currently it is not known how AD is caused. A new and exciting project here at NSCI is exploring this exact question. Using neural stem cells, we are investigating a novel hypothesis as to what causes the neurons in AD to start dying. We hope this information will lead to critical information on how AD is caused, and subsequently how it can be treated.

Our Unique Organization

The Neural Stem Cell Institute (NSCI) is dedicated to developing regenerative stem cell therapies for various diseases of the central nervous system (CNS): the brain, spinal cord and retina, regardless of the cause of injury. Led by MacArthur “Genius” Award winner Dr. Sally Temple who helped discover and define nervous system stem cells, NSCI is the first independent, non-profit stem cell research institute in the USA.